[go: up one dir, main page]

DK3504229T3 - REGULATION OF GENEPRESSION USING MANIPULATED NUCLEASES - Google Patents

REGULATION OF GENEPRESSION USING MANIPULATED NUCLEASES Download PDF

Info

Publication number
DK3504229T3
DK3504229T3 DK17844404.8T DK17844404T DK3504229T3 DK 3504229 T3 DK3504229 T3 DK 3504229T3 DK 17844404 T DK17844404 T DK 17844404T DK 3504229 T3 DK3504229 T3 DK 3504229T3
Authority
DK
Denmark
Prior art keywords
genepression
regulation
nucleases
manipulated
manipulated nucleases
Prior art date
Application number
DK17844404.8T
Other languages
Danish (da)
Inventor
Jeffrey C Miller
Edward J Rebar
Original Assignee
Sangamo Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Application granted granted Critical
Publication of DK3504229T3 publication Critical patent/DK3504229T3/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
DK17844404.8T 2016-08-24 2017-08-24 REGULATION OF GENEPRESSION USING MANIPULATED NUCLEASES DK3504229T3 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662378978P 2016-08-24 2016-08-24
US201762443981P 2017-01-09 2017-01-09
US201762545778P 2017-08-15 2017-08-15
PCT/US2017/048397 WO2018039440A1 (en) 2016-08-24 2017-08-24 Regulation of gene expression using engineered nucleases

Publications (1)

Publication Number Publication Date
DK3504229T3 true DK3504229T3 (en) 2021-12-20

Family

ID=61246246

Family Applications (1)

Application Number Title Priority Date Filing Date
DK17844404.8T DK3504229T3 (en) 2016-08-24 2017-08-24 REGULATION OF GENEPRESSION USING MANIPULATED NUCLEASES

Country Status (32)

Country Link
US (2) US10563184B2 (en)
EP (2) EP3504229B1 (en)
JP (1) JP7203014B2 (en)
KR (1) KR102572759B1 (en)
CN (1) CN110325635B (en)
AU (2) AU2017315406B2 (en)
BR (1) BR112019003100A2 (en)
CA (1) CA3034882A1 (en)
CL (1) CL2019000399A1 (en)
CO (1) CO2019001364A2 (en)
CR (1) CR20190098A (en)
DK (1) DK3504229T3 (en)
DO (1) DOP2019000031A (en)
EC (1) ECSP19017082A (en)
ES (1) ES2900317T3 (en)
HR (1) HRP20212025T1 (en)
HU (1) HUE056707T2 (en)
IL (1) IL264639B2 (en)
LT (1) LT3504229T (en)
MX (1) MX2019002207A (en)
MY (1) MY193234A (en)
NZ (1) NZ750938A (en)
PE (1) PE20190568A1 (en)
PH (1) PH12019500281A1 (en)
PL (1) PL3504229T3 (en)
PT (1) PT3504229T (en)
RS (1) RS62758B1 (en)
SG (2) SG11201901531TA (en)
SI (1) SI3504229T1 (en)
TN (1) TN2019000048A1 (en)
WO (1) WO2018039440A1 (en)
ZA (1) ZA201900765B (en)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
GB2568182A (en) 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
HRP20212025T1 (en) * 2016-08-24 2022-04-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018039448A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Engineered target specific nucleases
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
CA3043635A1 (en) * 2016-12-01 2018-06-07 Sangamo Therapeutics, Inc. Tau modulators and methods and compositions for delivery thereof
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
TW201839136A (en) 2017-02-06 2018-11-01 瑞士商諾華公司 Composition and method for treating hemochromatosis
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (en) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 Cytosine to guanine base editor
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP2020534795A (en) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ Methods and Compositions for Evolving Base Editing Factors Using Phage-Supported Continuous Evolution (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
AU2018364660B2 (en) * 2017-11-09 2022-05-19 Sangamo Therapeutics, Inc. Genetic modification of cytokine inducible SH2-containing protein (CISH) gene
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
BR112020015488A2 (en) 2018-02-08 2020-12-08 Sangamo Therapeutics, Inc. MODIFIED TARGET-SPECIFIC NUCLEASES
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
KR20210070344A (en) 2018-10-02 2021-06-14 상가모 테라퓨틱스, 인코포레이티드 Methods and compositions for modulation of tau protein
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College Methods and compositions for editing nucleotide sequences
CN113939586A (en) 2019-04-02 2022-01-14 桑格摩生物治疗股份有限公司 Methods of treating beta-thalassemia
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
BR112022022603A2 (en) 2020-05-08 2023-01-17 Broad Inst Inc METHODS AND COMPOSITIONS FOR SIMULTANEOUS EDITING OF BOTH DUAL-STRANDED NUCLEOTIDE TARGET SEQUENCE STRAINS
CN114149990A (en) * 2020-09-08 2022-03-08 甘李药业股份有限公司 Method for editing BCL11A gene in hematopoietic stem/progenitor cells
US11514747B2 (en) 2020-09-30 2022-11-29 Aristocrat Technologies, Inc. Latching assembly for a pushbutton of an electronic gaming machine
WO2023014839A1 (en) * 2021-08-03 2023-02-09 Sangamo Therapeutics, Inc. Methods for the treatment of sickle cell disease
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (123)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2681922C (en) 1994-01-18 2012-05-15 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
AU696455C (en) 1994-03-23 2006-03-02 Case Western Reserve University Compacted nucleic acids and their delivery to cells
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
EP0781331B1 (en) 1994-08-20 2008-09-03 Gendaq Limited Improvements in or relating to binding proteins for recognition of dna
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
AU746454B2 (en) 1998-03-02 2002-05-02 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
ATE309536T1 (en) 1999-12-06 2005-11-15 Sangamo Biosciences Inc METHODS OF USING RANDOMIZED ZINC FINGER PROTEIN LIBRARIES TO IDENTIFY GENE FUNCTIONS
EP2207032A1 (en) 2000-02-08 2010-07-14 Sangamo BioSciences, Inc. Cells expressing zinc finger protein for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
JP2002060786A (en) 2000-08-23 2002-02-26 Kao Corp Bactericidal antifouling agent for hard surfaces
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
EP1353941B1 (en) 2001-01-22 2013-03-13 Sangamo BioSciences, Inc. Modified zinc finger binding proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (en) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Zinc finger binding domain for CNN
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
ATE531796T1 (en) 2002-03-21 2011-11-15 Sangamo Biosciences Inc METHODS AND COMPOSITIONS FOR USING ZINC FINGER ENDONUCLEASES TO IMPROVE HOMOLOGOUS RECOMBINATION
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
AU2003298574B2 (en) 2002-09-05 2008-04-24 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
DE602005011943D1 (en) 2004-04-08 2009-02-05 Sangamo Biosciences Inc COMPOSITIONS FOR THE TREATMENT OF NEUROPATHIC AND NEURODEEGENERATIVE DISEASES
JP2008506359A (en) 2004-04-08 2008-03-06 サンガモ バイオサイエンシズ インコーポレイテッド Treatment of neuropathic pain with zinc finger protein
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
EP1877583A2 (en) 2005-05-05 2008-01-16 Arizona Board of Regents on behalf of the Unversity of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
US20090263900A1 (en) 2008-04-14 2009-10-22 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
CA2651494C (en) 2006-05-25 2015-09-29 Sangamo Biosciences, Inc. Engineered cleavage half-domains
AU2007267874B2 (en) 2006-05-25 2012-03-15 Sangamo Therapeutics, Inc. Methods and compositions for gene inactivation
JP2010500029A (en) 2006-08-11 2010-01-07 ダウ アグロサイエンシィズ エルエルシー Zinc finger nuclease-mediated homologous recombination
AU2008244473B2 (en) 2007-04-26 2013-06-20 Sangamo Therapeutics, Inc. Targeted integration into the PPP1R12C locus
US8585526B2 (en) 2007-06-18 2013-11-19 American Axle & Manufacturing, Inc. Vehicle driveline component having heat sink for increased heat rejection capabilities
CN107501370B (en) 2007-06-19 2021-06-18 路易斯安那州州立大学及农业机械学院管理委员会 Synthesis and use of anti-reverse phosphorothioate analogs of messenger RNA caps
EP2188384B1 (en) 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
WO2009054985A1 (en) 2007-10-25 2009-04-30 Sangamo Biosciences, Inc. Methods and compositions for targeted integration
DE102007056956B4 (en) 2007-11-27 2009-10-29 Moosbauer, Peter, Dipl.-Ing.(FH) Circuit for controlling the power supply of a consumer and method for operating a circuit
US9394531B2 (en) 2008-05-28 2016-07-19 Sangamo Biosciences, Inc. Compositions for linking DNA-binding domains and cleavage domains
EP2789691B1 (en) 2008-08-22 2018-08-08 Sangamo Therapeutics, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
DK2334794T3 (en) 2008-09-15 2017-02-20 Children's Medical Center Corp MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
ES2627552T3 (en) 2008-12-04 2017-07-28 Sigma Aldrich Company Genome editing in rats using zinc finger nucleases
EP3581197A1 (en) 2009-07-31 2019-12-18 ethris GmbH Rna with a combination of unmodified and modified nucleotides for protein expression
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
EP2504430A4 (en) 2009-11-27 2013-06-05 Basf Plant Science Co Gmbh Chimeric endonucleases and uses thereof
MX340102B (en) 2010-01-28 2016-06-24 The Children's Hospital Of Philadelphia * A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy.
ES2751916T3 (en) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Genomanipulated half-cleavages
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
CA3122219A1 (en) * 2010-04-16 2011-10-20 The Children's Hospital Corporation Sustained polypeptide expression from synthetic, modified rnas and uses thereof
SG185367A1 (en) 2010-04-26 2012-12-28 Sangamo Biosciences Inc Genome editing of a rosa locus using zinc-finger nucleases
WO2011139349A1 (en) 2010-05-03 2011-11-10 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
JP6208580B2 (en) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド Novel DNA binding protein and use thereof
JP6050230B2 (en) 2010-07-21 2016-12-21 サンガモ バイオサイエンシーズ， インコーポレイテッド Methods and compositions for modification of HLA loci
US9057057B2 (en) 2010-07-27 2015-06-16 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
GB201014169D0 (en) * 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
CN102358902B (en) 2011-04-02 2013-01-02 西南大学 Silkworm fibroin heavy-chain gene mutation sequence and mutation method and application
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA3099582A1 (en) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
HK1200871A1 (en) 2011-11-16 2015-08-14 Sangamo Therapeutics, Inc. Modified dna-binding proteins and uses thereof
CN103361328B (en) * 2012-03-30 2017-08-18 复旦大学 The zinc finger endonuclease of the provirus gene knockouts of HIV 1 of mediated integration and its preparation method and application
WO2013169802A1 (en) 2012-05-07 2013-11-14 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
HUE064187T2 (en) 2012-05-25 2024-02-28 Cellectis Procedure for modification of allogeneic and immunosuppressive-resistant T cells suitable for immunotherapy
AU2013308770B2 (en) * 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
JP2016500254A (en) 2012-12-05 2016-01-12 サンガモ バイオサイエンシーズ， インコーポレイテッド Methods and compositions for the regulation of metabolic diseases
EP2935319B1 (en) * 2012-12-18 2019-04-24 Novartis AG Production of therapeutic proteins in genetically modified mammalian cells
KR102271292B1 (en) 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
HK1223401A1 (en) * 2013-05-15 2017-07-28 桑格摩生物科学股份有限公司 Methods and compositions for treatment of a genetic condition
EP3591045B1 (en) 2013-08-28 2024-07-17 Sangamo Therapeutics, Inc. Compositions for linking dna-binding domains and cleavage domains
HRP20211706T1 (en) 2013-11-13 2022-02-04 The Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
CA2931848A1 (en) * 2013-12-09 2015-06-18 Sangamo Biosciences, Inc. Methods and compositions for genome engineering
US20170037431A1 (en) * 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
AU2015255877B2 (en) 2014-05-08 2020-03-26 Chdi Foundation, Inc. Methods and compositions for treating huntington's disease
US9816074B2 (en) * 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
EP3054007A1 (en) 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step
MX382223B (en) 2015-05-12 2025-03-13 Sangamo Therapeutics Inc NUCLEASE-MEDIATED REGULATION OF GENE EXPRESSION.
US9957501B2 (en) * 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CN108024544B (en) 2015-07-13 2022-04-29 桑格摩生物治疗股份有限公司 Delivery methods and compositions for nuclease-mediated genome engineering
WO2018039448A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Engineered target specific nucleases
HRP20212025T1 (en) 2016-08-24 2022-04-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
EP3551169A4 (en) 2016-12-09 2020-09-09 Sangamo Therapeutics, Inc. ADMINISTRATION OF TARGET-SPECIFIC NUCLEASES
US20190048060A1 (en) 2017-08-08 2019-02-14 Sangamo Therapeutics, Inc. Chimeric antigen receptor mediated cell targeting

Also Published As

Publication number Publication date
KR20190039530A (en) 2019-04-12
ZA201900765B (en) 2023-02-22
CO2019001364A2 (en) 2019-03-18
TN2019000048A1 (en) 2020-07-15
CN110325635B (en) 2023-12-26
ECSP19017082A (en) 2019-03-29
US10563184B2 (en) 2020-02-18
RS62758B1 (en) 2022-01-31
JP7203014B2 (en) 2023-01-12
BR112019003100A2 (en) 2019-07-09
IL264639B1 (en) 2023-09-01
MX2019002207A (en) 2019-07-15
CL2019000399A1 (en) 2019-04-26
KR102572759B1 (en) 2023-08-29
PL3504229T3 (en) 2022-04-19
AU2017315406B2 (en) 2021-04-01
EP3504229B1 (en) 2021-10-13
DOP2019000031A (en) 2019-09-30
AU2017315406A1 (en) 2019-03-14
HUE056707T2 (en) 2022-03-28
IL264639A (en) 2019-03-31
IL264639B2 (en) 2024-01-01
EP3504229A4 (en) 2020-02-12
PT3504229T (en) 2021-12-13
WO2018039440A1 (en) 2018-03-01
EP3995574A1 (en) 2022-05-11
PE20190568A1 (en) 2019-04-22
EP3504229A1 (en) 2019-07-03
AU2021204432B2 (en) 2022-12-01
CA3034882A1 (en) 2018-03-01
SG10201913315SA (en) 2020-02-27
SG11201901531TA (en) 2019-03-28
HRP20212025T1 (en) 2022-04-01
MY193234A (en) 2022-09-27
CN110325635A (en) 2019-10-11
US20200131490A1 (en) 2020-04-30
SI3504229T1 (en) 2022-04-29
AU2021204432A1 (en) 2021-07-29
US20190177709A1 (en) 2019-06-13
NZ750938A (en) 2023-07-28
PH12019500281A1 (en) 2019-08-05
ES2900317T3 (en) 2022-03-16
US11845965B2 (en) 2023-12-19
CR20190098A (en) 2019-04-30
JP2019524150A (en) 2019-09-05
LT3504229T (en) 2021-12-10

Similar Documents

Publication Publication Date Title
DK3504229T3 (en) REGULATION OF GENEPRESSION USING MANIPULATED NUCLEASES
DK3512850T3 (en) INHIBITORS OF THE MENIN-MLL INTERACTION
DK3468966T3 (en) INHIBITORS OF THE MENIN-MLL INTERACTION
DK3265563T3 (en) REGULATION OF GENE EXPRESSION BY APTAMER MEDIATED MODULATION OF ALTERNATIVE splicing
DK3116533T3 (en) DELETION OF DYSTROPHINGENEXON USING GENMANIPULATED NUCLEASES
DK3509581T3 (en) FORMULATIONS OF (R)-2-AMINO-3-PHENYLPROPYLCARBAMATE
DK3377059T3 (en) INHIBITORS OF CXCR2
DK3137169T3 (en) INHIBITORIES OF LYSIN-SPECIFIC DEMETHYLASE-1
DK3492593T3 (en) NUCLEASE MEDIATED REGULATION OF GENE EXPRESSION
DK3851537T5 (en) TREATMENT OF HYPERBILIRUBINAMIA
DK3102576T3 (en) DIHYDROPYRROLOPYRIDINE INHIBITORS OF ROR-GAMMA
DK3317400T3 (en) PROCEDURE FOR CRYO-PRESERVATION OF TUMOR-INFILRATING LYMPOCYTS
DK3294325T3 (en) ENHANCEMENT OF THE EFFECT OF CAR-MANIPULATED T CELLS USING NUCLEIC ACID VACCINATION
DK3180331T3 (en) POLYMORPHS OF SELINEXOR
DK3286311T3 (en) PROCEDURE FOR TREATING MALIGNITIES
DK3325443T3 (en) PROCEDURE FOR THE MANUFACTURE OF PRIDOPIDINE
DK3164380T3 (en) INHIBITORS OF LIGHT-SPECIFIC DEMETHYLASE-1
DK3506904T3 (en) Treatment of dementia
DK3261726T3 (en) COMPOSITION FOR TREATMENT OF ACNE
DK3474822T3 (en) FORMULATIONS OF BRINCIDOFOVIR
DK3307267T3 (en) TREATMENT OF MULTIPLE SHELLOSIS
DK3261661T3 (en) MENOTROPIN FOR THE TREATMENT OF INFERTILITY
DK3204352T3 (en) Inhibitors of lysine-gingipain
DK3511000T3 (en) CRYSTALLINIC FORM X2 OF GRAPIPRANT
DK3297619T3 (en) Therapeutic uses of L-4-chlorokynurenine